ICER White Paper – Rare Disease Drug Policy Reform, Drug Access, and Innovation
Despite the advancements in rare disease treatment, patients with many rare diseases still have extremely limited, if any, treatment options. [...]
ISPOR Webinar – Delivering Evidence-Based Access in Rare Diseases: The Challenges in SMA – April 5th
Rare diseases like spinal muscular atrophy (SMA) pose specific challenges for HTA approval, especially regarding data gaps resulting from small [...]
Rare Patient Voice LLC Expands its Reach to Australia and New Zealand
The market research patient recruitment firm Rare Patient Voice LLC has announced that it is expanding its patient and caregiver [...]
Rare Disease Clinical Outcome Assessment Consortium Announced by C-Path
The Critical Path Institute (C-Path) has launched its Rare Disease Clinical Outcome Assessment Consortium. The Consortium is a public-private collaborative [...]
RWD Advances Rare Disease R&D, Brings Hope to Patients
Patient registries are fast becoming a critical source of real world data (RWD), especially in rare disease research and development [...]
eBook on Rare Disease Therapies and Marketing Now Available
Rare Disease Advisor is offering a free eBook on rare disease (RD) trends and marketing efforts. The eBook details highly [...]
AllStripes Announces $50 Million in Series B Funding to Advance Rare Disease Research
AllStripes has raised $50 million in Series B Funding, led by Lux Capital. As a result, the organization has initiated [...]
Arsenal’s Value Demonstration Acquires Guidemark Health Following Recent BresMed Health Solutions and Cello Health Acquisitions
Arsenal Capital Partners has acquired Guidemark Health, following its acquisitions of Cello Health and BresMed Health Solutions last year. Arsenal’s [...]
Newly Launched Bespoke Gene Therapy Consortium Raises $76 Million to Advance Rare Disease Research
The Food and Drug Administration, the National Institutes of Health, and the Foundation for the Institutes of Health have announced [...]
Parexel VPs Discuss COVID-19, RWE, and New Challenges
Parexel’s Leanne Larson and Matthew Gordon shared their thoughts on the growing importance of real world evidence (RWE) in clinical [...]
Infographic Details Solutions to Cell and Gene Therapy Manufacturing Challenges
A newly published infographic, provided by Catalent, highlights some of the challenges associated with cell and gene therapies—and how to [...]
ICER’s Value Assessments Inform Pricing Decisions
Although new technologies have brought about novel therapies for previously unaddressed diseases, setting prices for these sometimes life-saving treatments remains [...]
ICON Clinical Trial Experts Discuss the Intricacies of Rare Disease Studies
Two clinical trial experts at ICON shared their thoughts on clinical trial design, including important considerations and strategies for rare [...]
Rare Disease Meeting Highlights FDA’s RDCA-DAP Research Resource
A recent rare disease meeting brought together more than 400 attendees to introduce the Rare Disease Cures Accelerator-Data and Analytics [...]
2021’s PBMI Conference Featured Lively Discussions on Specialty Drug Costs, Data Interoperability, and RWD
The Pharmacy Benefit Management Institute (PBMI) recently held its annual conference. This year, the hybrid format allowed for lively discussions [...]
FDA Launches Rare Disease Cures Accelerator-Data and Analytics Platform
Funded by the Food and Drug Administration, the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) has just been launched. [...]
Will the Death of Patient with ALS Prompt Biogen to Improve Access to Experimental Drugs?
Lisa Stockman Mauriello passed away earlier this month after an intense fight to use Biogen’s experimental drug for Amyotrophic Lateral [...]
Managed Entry Agreements Could Bring Affordability to Multibillion-Dollar Novel Gene Therapy Market
Cogentia’s Senior Analyst Mark Orchard discusses how managed entry agreements (MEAs) could become a major trend in improving access to [...]
The Rare Patient Voice and RWD Leader Clinakos Announce Partnership Intended to Improve Understanding of the Patient Experience
The Rare Patient Voice, an organization that recruits patients diagnosed with rare diseases, and real world data (RWD) company Clinakos [...]
ICER Announces Positive Early Results in Draft Evidence Report for Two Myasthenia Graves Therapies
The Institute for Clinical and Economic Review (ICER) has published its Draft Evidence Report on two drugs, eculizumaband efgartigimod, used [...]
UK Seeks to Advance Cost-Effective Rare Disease Research, Allocates $340 Million to New Innovative Medicines Fund
The UK will expand its Cancer Drugs Fund by creating a new Innovative Medicines Fund. The fund will promote research [...]
IQVIA Case Study Highlights Solutions in Identifying Rare Disease RWD
IQVIA has published a case study where researchers sought real world data (RWD) for patients with Focal Segmental Glomerulosclerosis. Using [...]
PharmacoEconomics Article Highlights Rare Disease Therapeutics Tied to Patient Outcomes
A recent publication considers how treatments for rare diseases are priced according to patient outcomes. The study aims to compare [...]
Updated FDA Guidance Recommends Inclusion of Patients with Incurable Cancers in Clinical Trials
The Food and Drug Administration (FDA) has issued draft guidance instructing clinical trial sponsors to include patients with incurable cancers [...]
PRMA Consulting Offers Regenerative Medicine and Rare Disease Market Access Insight
PRMA Consulting is offering a selection of case studies that reflect the challenges facing stakeholders when considering market access strategies [...]
